GtoPdb Ligand ID: 9295

Synonyms: emapalumab-lzsg | Gamifant® | NI-0501 | NI0501
emapalumab is an approved drug (FDA (2018))
Compound class: Antibody
Comment: Emapalumab (NI-0501) is a fully human monoclonal antibody that targets interferon gamma (IFNγ).
Annotated peptide sequences for this antibody are available from its IMGT/mAb-DB record. BLAST peptide anaylsis reveals identical matches with peptides claimed in patent WO2006109191, and in which NI-0501 is confirmed as an exemplary antibody [2].
No information available.
Summary of Clinical Use
Emapalumab initially received FDA orphan drug designation for the treatment of hemophagocytic lymphohistiocytosis (HLH). Link here to the FDA record for this designation.
Phase 2/3 clinical trial NCT01818492 evaluated emapalumab (NI-0501) in children with primary HLH, and a long-term follow-up study of HLH patients who were given emapalumab in an earlier clinical trial is ongoing (see NCT02069899). In November 2018, the FDA granted full approval for emapalumab's use as a treament option for adult and pediatric (newborn and older) patients with refractory, recurrent or progressive primary HLH or who are intolerant to conventional HLH therapy.
Mechanism Of Action and Pharmacodynamic Effects
Familial (primary) HLH is a life-threatening immune disorder characterised by excessive production of activated lymphocytes (T cells, natural killer cells, B cells and macrophages) and severely elevated cytokine production. This unchecked immune activation causes neurological problems, abnormalities of the heart, kidneys, and other organs and tissues, anemia, and clotting problems. To date anti-inflammatory drugs such as dexamethasone, methotrexate and cortisol (hydrocortisone) as well as cyclosporin A and etoposide have been used to reduce the number of immune cells in HLH patients. Anti-IFNγ therapy is being exploited for this indication, with the aim of providing more effective and less toxic treatments, as suggested by studies in animal models of HLH [3].
Clinical Trials
Clinical Trial ID Title Type Source Comment References
NCT01818492 A Study to Investigate the Safety and Efficacy of an Anti-IFNγ mAb in Children Affected by Primary Haemophagocytic Lymphohistiocytosis Phase 2/Phase 3 Interventional NovImmune SA
NCT02069899 Long-term Follow-up of HLH Patients Who Received Treatment With NI-0501, an Anti-interferon Gamma Monoclonal Antibody N/A Interventional NovImmune SA
External links